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Results for “AMN”

82 trials

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Showing 20 of 82 results

Not applicableEnded earlyNCT04008849
What this trial is testing

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456

Who this might be right for
Inherited Metabolic Disorders (IMD)
Magenta Therapeutics, Inc. 3
Not applicableStudy completedNCT02952482
What this trial is testing

Newborn Screening for Adrenoleukodystrophy

Who this might be right for
Adrenoleukodystrophy
National Taiwan University Hospital 45,796
Not applicableLooking for participantsNCT05443906
What this trial is testing

Home Exercise for Individuals with Neurodegenerative Disease

Who this might be right for
Neurodegenerative DiseasesLeukodystrophyAtaxia+5 more
Hugo W. Moser Research Institute at Kennedy Krieger, Inc. 30
Large-scale testing (Phase 3)Study completedNCT01896102
What this trial is testing

The Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

Who this might be right for
Cerebral Adrenoleukodystrophy (CALD)
Genetix Biotherapeutics Inc. 32
Testing effectiveness (Phase 2)Study completedNCT00762632
What this trial is testing

Combination of Nilotinib (AMN107) and RAD001 in Patients With Acute Myeloid Leukemia

Who this might be right for
Acute Myeloid Leukemia
Technical University of Munich 40
Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Not applicableLooking for participantsNCT05939232
What this trial is testing

Registry of X-linked Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
Beijing Tiantan Hospital 200
Not applicableNo Longer AvailableNCT02233257
What this trial is testing

Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
University of Minnesota
Not applicableStudy completedNCT00538109
What this trial is testing

An Open-Label, Multicenter, Expanded Access Study of Oral AMN 107 in Adult Patients With Imatinib (Glivec®/Gleevec®_ - Resistant or Intolerant Chronic Myeloid Leukemia in Blast Crisis, Accelerated Phase or Chronic Phase

Who this might be right for
Leukemia, Myeloid, Chronic
King Faisal Specialist Hospital & Research Center 8
Not applicableStudy completedNCT00004450
What this trial is testing

Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy

Who this might be right for
Adrenoleukodystrophy
FDA Office of Orphan Products Development 60
Large-scale testing (Phase 3)Ended earlyNCT00654433
What this trial is testing

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

Who this might be right for
Inherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more
Aldagen 40
Not applicableLooking for participantsNCT04675749
What this trial is testing

Quality of Life in Women with X-linked Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
Leipzig University Medical Center 200
Not applicableNot Yet RecruitingNCT05911919
What this trial is testing

Validation of a Prognostic Biomarker Using Brain Diffusion MRI in X-linked Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
Assistance Publique - Hôpitaux de Paris 200
Not applicableNo Longer AvailableNCT00302016
What this trial is testing

Expanded Access Program of AMN107 in Imatinib-resistant or Intolerant Adult Patients With Chronic Myeloid Leukemia

Who this might be right for
Chronic Myeloid Leukemia
Novartis Pharmaceuticals
Testing effectiveness (Phase 2)UnknownNCT02559830
What this trial is testing

Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy

Who this might be right for
Metachromatic LeukodystrophyAdrenoleukodystrophy
Shenzhen Second People's Hospital 50
Testing effectiveness (Phase 2)Study completedNCT01043640
What this trial is testing

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Who this might be right for
MucopolysaccharidosisHurler SyndromeHunter Syndrome+10 more
Masonic Cancer Center, University of Minnesota 46
Large-scale testing (Phase 3)UnknownNCT03143049
What this trial is testing

Pomalidomide-Cyclophosphamide-Dexamethasone (PCD) Versus Pomalidomide-Dexamethasone (PD) in Relapse or Refractory Myeloma

Who this might be right for
Relapse Multiple Myeloma
National University Hospital, Singapore 120
Not applicableStudy completedNCT02699190
What this trial is testing

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Who this might be right for
LeukodystrophyWhite Matter Disease4H Syndrome+61 more
Children's Hospital of Philadelphia 236
Not applicableEnded earlyNCT02476682
What this trial is testing

Non-invasive Risk Stratification of CR AMN/SSP

Who this might be right for
Adenomatous Polyps
Professor Michael Bourke 205
Early research (Phase 1)Study completedNCT03500627
What this trial is testing

Evaluate the Safety, Tolerability, and Pharmacokinetics of OP-101 After Intravenous Administration in Healthy Volunteers

Who this might be right for
Healthy
Orpheris, Inc. 8
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